An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

NCT00882921 | Completed Results

• 1 other identifier: HGT-ELA-042
• Study Type: observational
• Target: 26
• Locations: 3 countries
• Sites: 6

Brief Summary

The objective of this study is to evaluate the effect of anti-idursulfase antibodies on idursulfase safety (measured by infusion related adverse events) between patients who develop anti-idursulfase antibodies and patients who do not after long-term idursulfase enzyme replacement therapy (ERT).

Conditions

Hunter Syndrome

Keywords

MPS 2; MPSII; hunter's disease treatment; enzyme replacement therapy; hunter's disease; iduronate 2 sulfatase; mps diagnosis; mps symptoms; iduronate sulfatase; MPS II treatment; chronic ear infection; hunters syndrome; mps ii therapy; MPS2; hunter's syndrome; Hunter syndrome; elaprase; lysosomal storage disease; hunter syndrome therapy; mps society; hunter syndrome treatment; hunter's syndrome treatment; mucopolysaccharides; mps ii; idursulfase; hunters disease; enlarged adenoids; ert treatment; hunter disease; lysosomal storage disorder

Outcome Measures

Primary Outcomes (1)

• Infusion-Related Adverse Event (IRAE) Rates Between IgG Anti-idursulfase Antibody Positive (Ab+) and Anti-idursulfase IgG Antibody Negative (Ab-) Patients

  The primary analysis of how presence of antibodies affected IRAE rates was performed based on a negative binomial regression model. This was done to account for potentially differential follow-up time between antibody groups.

  Baseline to 109 Weeks

Secondary Outcomes (1)

• Change From Baseline in uGAG Levels to 109 Weeks

  Baseline to 109 Weeks

Study Arms (1)

Elaprase

Idursulfase 0.5 mg/kg Weekly

Biological: Idursulfase

Interventions

Idursulfase BIOLOGICAL

Patients received idursulfase as prescribed by their physician following locally approved prescribing information. Patients will not be provided idursulfase by Shire Human Genetic Therapies, Inc. or the HOS.

Also known as: Elaprase

Elaprase

Eligibility Criteria

• Age: 5 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with Hunter syndrome

You may qualify if:

• Patients must meet all of the following criteria to be considered eligible for enrollment:
• The patient is male and enrolled in the HOS (i.e., meets the entry criteria of a documented diagnosis of Hunter syndrome)
• The patient is ≥ 5 years-old
• The patient is on idursulfase treatment or scheduled to begin idursulfase treatment within 30 days of study enrollment
• The patient, patient's parent(s), or patient's legally authorized guardian must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient, patient's parent(s), or patient's legally authorized guardian.

You may not qualify if:

• Patients who meet any of the following criteria are not eligible for this study:
• The patient has received biologic/ERT products other than idursulfase, or other investigational product(s) for any reason within 30 days prior to study entry.
• The patient has a life expectancy of \< 2 years
• The patient is unable to comply with the protocol, e.g., has a clinically relevant medical condition making implementation of the protocol difficult; has an uncooperative attitude; is unable to return for safety evaluations; or is otherwise unlikely to complete the study, as determined by the Investigator.

Sponsors & Collaborators

• Shire: lead

Study Sites (6)

Children's Hospital & Research Center Oakland

Oakland, California, 94609, United States

Location

Children's Hospitals and Clinics of Minnesota, Division of Genetics

Minneapolis, Minnesota, 55404, United States

Location

Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica

Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

Location

Birmingham Children's Hospital

Birmingham, B46NH, United Kingdom

Location

Great Ormond Street Hospital

London, WC1N 3JH, United Kingdom

Location

Central Manchester University Hospitals, St. Mary's Hospital

Manchester, M139WL, United Kingdom

Location

Related Publications (1)

• Giugliani R, Harmatz P, Jones SA, Mendelsohn NJ, Vellodi A, Qiu Y, Hendriksz CJ, Vijayaraghavan S, Whiteman DA, Pano A. Evaluation of impact of anti-idursulfase antibodies during long-term idursulfase enzyme replacement therapy in mucopolysaccharidosis II patients. Mol Genet Metab Rep. 2017 Feb 21;12:2-7. doi: 10.1016/j.ymgmr.2017.01.014. eCollection 2017 Sep.

  PMID: 28243577 RESULT

Biospecimen

Retention: SAMPLES WITHOUT DNA

Blood and urine

MeSH Terms

Conditions

Mucopolysaccharidosis II; Sudden Infant Death; Lysosomal Storage Diseases

Interventions

idursulfase

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Death, Sudden; Death; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Infant Death

Results Point of Contact

• Title: Study Director
• Organization: Shire

ClinicalTransparency@shire.com

+1 866 842 5335

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 16, 2009
• First Posted: April 17, 2009
• Study Start: October 14, 2008
• Primary Completion: February 8, 2013
• Study Completion: February 8, 2013
• Last Updated: June 8, 2021
• Results First Posted: July 30, 2014

Record last verified: 2021-05

Locations

BR (1); GB (3); US (2)